Fri.Jul 12, 2024

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US and UK fast track designations for Parkinson’s gene therapy

Drug Discovery World

The US Food and Drug Administration (FDA) has granted Fast Track Designation for Asklepios BioPharmaceutical’s (AskBio) AB-1005 which is being developed for moderate Parkinson’s disease. AB-1005 has also been awarded the innovative medicine designation, the Innovation Passport by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of Parkinson’s disease.

Therapies 147
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Synthesis and cytotoxic evaluation of heterocyclic compounds by vinylic substitution of ketene dithioacetals

Chemical Biology and Drug Design

In this work, double vinylic substitution reactions in ketene dithioacetals were used to synthesize a small library of heterocycles derivatives. The 19 derivatives were evaluated with breast and ovarian cancer cells. Two benzoxazoles, with calculated physicochemical properties within the range of drug-like compounds, presented good potency and selectivity.

Drugs 100
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Increase in diabetes research fuelled by GLP-1s like Ozempic

Drug Discovery World

Covid-19 is no longer in the top five most studied indications, replaced by type 2 diabetes, according to Phesi’s mid-year analysis of clinical trials. The analysis of 66,935 clinical trials reveals that the top five most studied diseases are now breast cancer, solid tumours, stroke, prostate cancer and type 2 diabetes. The mid-year analysis indicates breast cancer could be the most researched disease area for four-years running, after topping the table last year.

Research 147
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Novel phenylpiperazine derivatives as potent transient receptor potential vanilloid 1 antagonists

Chemical Biology and Drug Design

The preferred compound L-21 , a novel phenylpiperazine derivative, was identified as a potent transient receptor potential vanilloid 1 antagonist. Abstract Transient receptor potential vanilloid 1 (TRPV1) is a non-selective cation channel, which is considered a highly validated target for pain perception. Repeated activation with agonists to desensitize receptors or use the antagonists can both exert analgesic effects.

Research 100
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How Machine Learning Drives Clinical Trial Efficiency

Clinical trial data management is increasingly challenging as studies grow in complexity. Quickly accessing and analyzing study data is vital for assessing trial progress and patient safety. In this paper, we explore real-time data access and analysis for proactive study management. We investigate using adverse event (AE) data to monitor safety and discuss a clinical analytics platform that supports collaboration and data review workflows.

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First-in-human vaccine trial for deadly Marburg virus

Drug Discovery World

Scientists at the University of Oxford in the UK have launched a new clinical trial to test a vaccine to protect against “devastating” Marburg virus. This is the first-in-human trial of the ChAdOx1 Marburg vaccine, which has been developed and manufactured by researchers at the University of Oxford. Marburg virus is a highly virulent disease in the same family as Ebola that is fatal in up to 88% of cases and has no approved vaccines or treatments.

Virus 147
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Insight into one of life's earliest ancestors revealed in new study

Science Daily: Pharmacology News

Researchers have shed light on Earth's earliest ecosystem, showing that within a few hundred million years of planetary formation, life on Earth was already flourishing.

Research 117

More Trending

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Fake Botox Shots Land 13 Women in Hospital

Drugs.com

FRIDAY, July 12, 2024 -- Seventeen women in nine states have fallen ill after getting fake Botox shots, with 13 of them landing in the hospital and one requiring a ventilator, a new report warns.In the report, published Thursday by the U.S. Centers.

Hospitals 112
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How the 'heart and lungs' of a galaxy extend its life

Science Daily: Pharmacology News

Galaxies avoid an early death because they have a 'heart and lungs' which effectively regulate their 'breathing' and prevent them growing out of control, a new study suggests. If they didn't, the universe would have aged much faster than it has and all we would see today is huge 'zombie' galaxies teeming with dead and dying stars. That's according to a new study that investigates one of the great mysteries of the Universe -- why galaxies are not as large as astronomers would expect.

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Thousands of Hospital Patients in Oregon May Have Been Exposed to Hepatitis, HIV

Drugs.com

FRIDAY, July 12, 2024 -- After an anesthesiologist may have exposed thousands of people treated at several hospitals in Oregon to hepatitis and HIV, those patients are being advised to get tested for the diseases.Two health care providers in.

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At the Breaking Point: The Unsustainable Future of Government Drug Discount Programs

Drug Channels

Today’s guest post comes from Gavin Magaha, Senior Director of External Affairs and Policy at Kalderos. Gavin discusses the three key reasons for duplicate discounts between the 340B Drug Pricing Program and the Medicaid Drug Rebate Program. He then explains Oregon’s novel approach to fixing the problem. To learn more, register for Kalderos’ August 21, 2024, webinar: Revolutionizing Government Discount Programs: Why Traditional Methods Fail.

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Deliver Fast, Flexible Clinical Trial Insights with Spotfire

Clinical research has entered a new era, one that requires real-time analytics and visualization to allow trial leaders to work collaboratively and to develop, at the click of a mouse, deep insights that enable proactive study management. Learn how Revvity Signals helps drug developers deliver clinical trial data insights in real-time using a fast and flexible data and analytics platform to empower data-driven decision-making.

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Bristol Myers’ leukemia blockbuster set to face first generic rival

BioPharma Drive: Drug Pricing

Patent litigation has opened the door to generic copies of Bristol Myers’ Sprycel arriving in the U.S. as soon as September.

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Four in 10 Cancer Cases, Nearly Half of Cancer Deaths Linked to Lifestyle

Drugs.com

FRIDAY, July 12, 2024 -- Nearly half of cancer deaths and 4 of 10 cases of cancer are linked to a person’s lifestyle, a new study says.Cigarette smoking remains the biggest cancer risk, contributing to 30% of cancer deaths and 20% of cancer cases, r.

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With gene editing, mice with a form of inherited deafness can hear again

Science Daily: Pharmacology News

Researchers have used gene editing to restore hearing in adult mice with a type of inherited hearing loss. They showed that shutting down a damaged copy of a gene called a microRNA (miRNA) enabled the animals to regain hearing. The approach may eventually lead to potential treatments for inherited hearing loss in people.

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Gun Laws Help Lower Suicides, not Murders, Among Children

Drugs.com

FRIDAY, July 12, 2024 -- Restrictive gun laws can decrease suicide rates among children and teenagers, but they don’t seem to lower their risk of being murdered, a new study says.States with laws requiring safe storage of firearms and mandatory w.

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Clinical Data Like You´ve Never Seen It Before: Why Spotfire Is the Leading Tool for Clinical Analytics

Clinical development organizations face a wide array of challenges when it comes to data, many of which can impact the operational effectiveness of their clinical trials. In this whitepaper, experts from Revvity Signals explore how solutions like TIBCO® Spotfire® enable better, more streamlined studies. The whitepaper also features a success story from Ambrx, a leading biopharmaceutical company, detailing how it has leveraged Spotfire to tackle data quality and collaboration challenges in clinic

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All stem cell therapies are not created equal

Science Daily: Pharmacology News

Researchers have found that two of the most frequently administered stem cell therapies, which are often used interchangeably, actually contain completely different types of cells.

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Having Diabetes Raises Risk of Failure With Spinal Fusion Surgery

Drugs.com

FRIDAY, July 12, 2024 -- Diabetes can make lumbar spinal fusion surgery much more likely to fail, a new study says.People with diabetes are nearly three times more likely to have their vertebrae fail to properly heal and fuse together, what.

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Leqembi sales inch higher; Spark’s pivot leads to layoffs

BioPharma Drive: Drug Pricing

Sales data suggest forecasts for the Alzheimer’s drug may be in reach. Elsewhere, Ipsen is licensing an ADC and Novartis is closing a San Diego site.

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Cigarettes Worse Than Vaping for Secondhand Smoke Exposure

Drugs.com

FRIDAY, July 12, 2024 -- Secondhand smoke from cigarettes is much worse than that from vaping, in terms of exposing children to nicotine, a new study finds.Children exposed to vaping indoors absorb less than one-seventh the amount of nicotine as.

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New tentative approval for Alembic drug dabigatran etexilate mesylate

Drug Patent Watch

Dabigatran etexilate mesylate is the generic ingredient in two branded drugs marketed by Alkem Labs Ltd, Apotex, Hetero Labs Ltd Iii, and Boehringer Ingelheim and, and is included in five NDAs.

Drugs 59
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Litigants pile into ethylene oxide rule review at D.C. Circuit

Agency IQ

BY WALKER LIVINGSTON, ESQ Litigation over the EPA’s new ethylene oxide sterilization rule has attracted a diverse array of petitioners challenging or supporting the agency’s regulation. The parties’ requests to intervene in the case, in addition to statements on the issues they plan to bring, provide an indication of the potential litigation to come.

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Embarking on a Journey: Universal Design in Health Systems

Perficient: Drug Development

I am thrilled to announce the launch of our new blog series focused on Universal Design and its application in health systems. This series will explore how Universal Design principles can revolutionize healthcare environments, making them more accessible, inclusive, and efficient for everyone. From hospitals to clinics, and digital health tools to patient interactions, we will delve into the myriad ways that thoughtful design can enhance the health and well-being of all individuals.

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FDA gives companies leeway to go after misinformation, but not free reign

Agency IQ

BY LAURA DIANGELO, MPH In a new revised draft guidance document, the FDA says it will allow life sciences companies to address misinformation about their products that is posted online. While the policy is similar in some respects to one first created by the FDA in 2014, the latest version is considerably more detailed regarding what it will – and won’t – allow companies to do.

FDA 59
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The Crucial Role of Universal Design in Health Systems- Universal Design in Health Systems-1

Perficient: Drug Development

As we delve into the application of Universal Design principles within health systems, it’s essential to understand why this approach matters so profoundly. Universal Design aims to create environments that are accessible, usable, and beneficial for everyone, regardless of age, ability, or background. When applied to health systems, these principles can transform the patient experience, enhance healthcare outcomes, and foster inclusivity.

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New EC report dives deep to better understand shortages of critical medicines

Agency IQ

BY KIRSTEN MESSMER, PHD, RAC Medicines shortages have been a major concern for public health for several years. Now, in a new report focused on critical medicines, the European Commission has taken a deeper look at the causes of shortages among a subset of critical medicines that have recently been in shortage. Its findings indicate that some problems are likely to be intractable without considerable effort on the part of policymakers.

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Older Adults More Stressed About Inflation, Hold Off on Health Care

Drugs.com

FRIDAY, July 12, 2024 -- Financial stress persists even though rising inflation rates have eased, and the group feeling the pinch most acutely is in its peak earning years, a new poll shows."Our biggest surprise from this poll is that the age group.

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EPA grants petition on PFAS in fluorinated containers

Agency IQ

BY WALKER LIVINGSTON, ESQ Following a loss at the Fifth Circuit, the EPA has a new plan to regulate PFAS in fluorinated plastic containers: traditional Toxic Substances Control Act Section 6 rulemaking. The road to a rule will be longer one, but the EPA’s granting a petition to regulate the substances is the first step towards its wider regulation scheme for PFAS in plastics.

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Heart Surgery for Older Women Is Often Risky Business, Study Finds

Drugs.com

FRIDAY, July 12, 2024 -- Older women are more likely than older men to die following heart bypass surgery, possibly due to the quality of the hospitals where they undergo the procedure, a new study says.Women older than 65 are 26% more likely than.

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The 53 regulations that FDA is currently working on

Agency IQ

BY ALEXANDER GAFFNEY, MS, RAC The FDA on Friday unveiled its much-anticipated Spring 2024 Unified Agenda, a document outlining the regulations the agency plans to release in 2024 and beyond. While the document is mostly familiar to long-time readers of the Unified Agenda, it does include seven new proposed rules and the new target publication dates for several major regulatory actions.

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Black Patients Diagnosed With Huntington's Later Than Whites

Drugs.com

FRIDAY, July 12, 2024 -- Black patients with Huntington’s disease receive their diagnoses an average of one year later than white people with the incurable genetic disorder, a new study shows.Early diagnosis is essential to help patients get proper.

Disease 52
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Perficient Colleague Attains Champion Status

Perficient: Drug Development

Databricks has recognized David Callaghan as a Partner Champion. As the first Perficient colleague to receive inclusion in the program, David is paving the way for others to get their footing with the partner. Program Overview To be a Databricks Partner champion, one must: Display Thought Leadership Harness Technical Expertise Become Community Leader Demonstrate Innovation Individuals who show promise and interest are approached with an intensive multi-step program that leads to becoming an offi

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Nearly 1 in 10 Pregnant Women Who Get COVID Develop Long COVID

Drugs.com

FRIDAY, July 12, 2025 -- Almost 10% of women who get COVID during pregnancy develop long-lasting symptoms, and a new study suggests doctors may be overlooking them."I doubt most obstetric clinicians are as aware of Long COVID as perhaps we should.

Doctors 52
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Article FDA Thank You FDA gives companies leeway to go after misinformation, but not free reign

Agency IQ

FDA gives companies leeway to go after misinformation, but not free reign In a new revised draft guidance document, the FDA says it will allow life sciences companies to address misinformation about their products that is posted online. While the policy is similar in some respects to one first created by the FDA in 2014, the latest version is considerably more detailed regarding what it will – and won’t – allow companies to do.

FDA 40
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New Report Calls for More Research on Women's Health Issues

Drugs.com

FRIDAY, July 12, 2024 -- A new report finds research is sorely lacking on how chronic illnesses affect women, and it urged government agencies to do more to investigate how these diseases strike women differently.The National Academies of Sciences.

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Article Periodic Thank You EPA grants petition on PFAS in fluorinated containers

Agency IQ

EPA grants petition on PFAS in fluorinated containers Following a loss at the Fifth Circuit, the EPA has a new plan to regulate PFAS in fluorinated plastic containers: traditional Toxic Substances Control Act Section 6 rulemaking. The road to a rule will be longer one, but the EPA’s granting a petition to regulate the substances is the first step towards its wider regulation scheme for PFAS in plastics.